05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
Pharmaceutical preparations and substances for the treatment
of cardiovascular diseases and disorders; pharmaceutical
preparations and substances for the treatment of bone
diseases and disorders; pharmaceutical preparations and
substances for the treatment of pathological calcification;
pharmaceutical preparations and substances for the treatment
of blood plasma pyrophosphate deficiency disorders;
pharmaceutical preparations and substances for the treatment
of kidney diseases and disorders.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
Pharmaceutical preparations and substances for the treatment
of cardiovascular diseases and disorders; pharmaceutical
preparations and substances for the treatment of bone
diseases and disorders; pharmaceutical preparations and
substances for the treatment of pathological calcification;
pharmaceutical preparations and substances for the treatment
of blood plasma pyrophosphate deficiency disorders;
pharmaceutical preparations and substances for the treatment
of kidney diseases and disorders.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
(1) Pharmaceutical preparations and substances for the treatment of cardiovascular diseases and disorders; pharmaceutical preparations and substances for the treatment of bone diseases and disorders; pharmaceutical preparations and substances for the treatment of pathological calcification; pharmaceutical preparations and substances for the treatment of blood plasma pyrophosphate deficiency disorders; pharmaceutical preparations and substances for the treatment of kidney diseases and disorders.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
(1) Pharmaceutical preparations and substances for the treatment of cardiovascular diseases and disorders; pharmaceutical preparations and substances for the treatment of bone diseases and disorders; pharmaceutical preparations and substances for the treatment of pathological calcification; pharmaceutical preparations and substances for the treatment of blood plasma pyrophosphate deficiency disorders; pharmaceutical preparations and substances for the treatment of kidney diseases and disorders.
5.
TREATMENT OF ENPP1 DEFICIENCY AND ABCC6 DEFICIENCY
The present disclosure provides, among other things, specific doses of an ENPP1 agent for in vivo treatment of an ENPP1 deficiency, such as for treatment of Generalized Arterial Calcification of Infancy (GACI), Autosomal Recessive Hypophosphatemic Rickets 2 (ARHR2), and other diseases resulting from pathological calcification, ENPP1 deficiency, ABCC6 deficiency such as diseases or disorders involving ectopic calcification of soft tissue in a subject.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
Pharmaceutical preparations and substances for the treatment of cardiovascular diseases and disorders; Pharmaceutical preparations and substances for the treatment of bone diseases and disorders; Pharmaceutical preparations and substances for the treatment of pathological calcification; Pharmaceutical preparations and substances for the treatment of blood plasma pyrophosphate deficiency disorders; Pharmaceutical preparations and substances for the treatment of kidney diseases and disorders
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
Pharmaceutical preparations and substances for the treatment of cardiovascular diseases and disorders; Pharmaceutical preparations and substances for the treatment of bone diseases and disorders; Pharmaceutical preparations and substances for the treatment of pathological calcification; Pharmaceutical preparations and substances for the treatment of blood plasma pyrophosphate deficiency disorders; Pharmaceutical preparations and substances for the treatment of kidney diseases and disorders
8.
LYOPHILIZED ENPP1 POLYPEPTIDE FORMULATIONS AND USES THEREOF
In certain aspects, the present invention provides novel lyophilized formulations of ENPP1 polypeptides, as well as methods for using such lyophilized formulations and reconstituted formulations of ENPP1 to treat an indication associated an ENPP1 deficiency. The formulations and methods provided herein are useful in treating diseases associated with an ENPP1 deficiency such as pathological calcification or pathological ossification.
in vivoin vivo treatment of an ENPP1 deficiency, such as for treatment of Generalized Arterial Calcification of Infancy (GACI), Hypopyrophosphatemia, Autosomal Recessive Hypophosphatemic Rickets 2 (ARHR2), and other diseases resulting from pathological calcification, ENPP1 deficiency, ABCC6 deficiency such as diseases or disorders involving ectopic calcification of soft tissue in a subject of age 1-12.
A61P 13/12 - Médicaments pour le traitement des troubles du système urinaire des reins
A61P 19/04 - Médicaments pour le traitement des troubles du squelette des troubles non-spécifiques du tissu conjonctif
A61P 9/00 - Médicaments pour le traitement des troubles du système cardiovasculaire
10.
METHODS OF IMPROVING CARDIOVASCULAR FUNCTION AND TREATING CARDIOVASCULAR DISEASE USING A RECOMBINANT ECTONUCLEOTIDE PYROPHOSPHATASE PHOSPHODIESTERASE (NPP1)
The present invention provides methods for improving cardiovascular function in a human patient (e.g., reducing hypertension), as well as methods of treating a cardiovascular disease, by administering a recombinant human soluble ectonucleotide pyrophosphatase phosphodiesterase (hsNPP1), active fragment or fusion protein thereof.
C12N 9/16 - Hydrolases (3.) agissant sur les liaisons esters (3.1)
A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
A61P 9/10 - Médicaments pour le traitement des troubles du système cardiovasculaire des maladies ischémiques ou athéroscléreuses, p. ex. médicaments antiangineux, vasodilatateurs coronariens, médicaments pour le traitement de l'infarctus du myocarde, de la rétinopathie, de l'insuffisance cérébro-vasculaire, de l'artériosclérose rénale
The present disclosure provides, among other things, specific doses of an ENPP1 agent for in vivo treatment of an ENPP1 deficiency, such as for treatment of Generalized Arterial Calcification of Infancy (GACI), Autosomal Recessive Hypophosphatemic Rickets 2 (ARHR2), and other diseases resulting from pathological calcification, ENPP1 deficiency, ABCC6 deficiency such as diseases or disorders involving ectopic calcification of soft tissue in a subject.
The present disclosure provides, among other things, vectors for expression of ENPP1 or ENPP3 in vivo and methods for the treatment of diseases of calcification and ossification in a subject.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61P 19/08 - Médicaments pour le traitement des troubles du squelette des maladies osseuses, p. ex. rachitisme, maladie de Paget
The present disclosure provides compositions and methods for treating allograft vasculopathy, for treating Moyamoya Diseases (MMD) and Moyamoya Syndrome (MMS), for treating inhibiting or preventing unwanted intimal proliferation in a subject by administering an ectonucleotide pyrophosphatase phosphodiesterase-1 (ENPP1) agent or an ectonucleotide pyrophosphatase phosphodiesterase-3 (ENPP3).
A61K 47/68 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un anticorps, une immunoglobuline ou son fragment, p. ex. un fragment Fc
The present disclosure provides compositions and methods for treating Peripheral Artery Diseases in a subject by administering an ectonucleotide pyrophosphatase phosphodiesterase-1 (ENPP1) agent or an ectonucleotide pyrophosphatase phosphodiesterase-3 (ENPP3).
A61P 9/10 - Médicaments pour le traitement des troubles du système cardiovasculaire des maladies ischémiques ou athéroscléreuses, p. ex. médicaments antiangineux, vasodilatateurs coronariens, médicaments pour le traitement de l'infarctus du myocarde, de la rétinopathie, de l'insuffisance cérébro-vasculaire, de l'artériosclérose rénale
The present disclosure provides compositions and methods for treating vascular smooth muscle cell proliferation in a subject that does not have a deficiency of ectonucleotide pyrophosphatase phosphodiesterase-1 (ENPP1) resulting in a pathological disease of calcification or ossification by administering an ENPP1 agent or an ENPP3 agent.
The present disclosure provides, among other things, specific doses of an ENPP1 agent for in vivo treatment of an ENPP1 deficiency, such as for treatment of Generalized Arterial Calcification of Infancy (GACI), Autosomal Recessive Hypophosphatemic Rickets 2 (ARHR2), and other diseases resulting from pathological calcification, ENPP1 deficiency, ABCC6 deficiency such as diseases or disorders involving ectopic calcification of soft tissue in a subject.
C12N 9/16 - Hydrolases (3.) agissant sur les liaisons esters (3.1)
A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
The present invention provides a method of treating NPP1 deficiency or NPP1-associated disease such as idiopathic infantile arterial calcification (IIAC), pseudoxanthoma elasticum, vascular calcification in chronic kidney disease (VCCKD), insulin resistance, hypophosphatemic rickets, myocardial ischemia, joint calcification, angioid streaks, and ossification of the posterior longitudinal ligament of the spine. The present invention provides a method for treating tissue calcification by administering soluble NPP1 to produce a transient increase in serum pyrophosphate levels.
The present invention provides a novel fusion polypeptide containing a catalytic domain of NPP1 fused to a targeting moiety, nucleic acids encoding the fusion polypeptide, a vector containing the nucleic acid integrated thereinto, a host cell transformed with the vector and pharmaceutical compositions comprising the fusion polypeptide.
In certain aspects, the present invention provides novel lyophilized formulations of ENPP1 polypeptides, as well as methods for using such lyophilized formulations and reconstituted formulations of ENPP1 to treat an indication associated an ENPP1 deficiency. The formulations and methods provided herein are useful in treating diseases associated with an ENPP1 deficiency such as pathological calcification or pathological ossification.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
A61K 38/00 - Préparations médicinales contenant des peptides
C12Q 1/6883 - Produits d’acides nucléiques utilisés dans l’analyse d’acides nucléiques, p. ex. amorces ou sondes pour les maladies provoquées par des altérations du matériel génétique
20.
TREATMENT OF DISEASES INVOLVING DEFICIENCY OF ENPP1 OR ENPP3
The present disclosure provides, among other things, vectors for expression of ENPP1 or ENPP3 in vivo and methods for the treatment of diseases of calcification and ossification in a subject.
A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
A61P 13/12 - Médicaments pour le traitement des troubles du système urinaire des reins
C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
The present invention provides a method of treating myointimal proliferation by administering a recombinant human soluble ectonucleotide pyrophosphatase phosphodiesterase (hsNPP1), active fragment or fusion protein thereof.
The present disclosure provides, among other things, specific doses of an ENPP1 agent for in vivo treatment of an ENPP1 deficiency, such as for treatment of Generalized Arterial Calcification of Infancy (GACI), Autosomal Recessive Hypophosphatemic Rickets 2 (ARHR2), and other diseases resulting from pathological calcification, ENPP1 deficiency, ABCC6 deficiency such as diseases or disorders involving ectopic calcification of soft tissue in a subject.
A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
C12Q 1/6883 - Produits d’acides nucléiques utilisés dans l’analyse d’acides nucléiques, p. ex. amorces ou sondes pour les maladies provoquées par des altérations du matériel génétique
24.
COMPOSITIONS AND METHODS FOR TREATING ALLOGRAFT VASCULOPATHY, MOYAMOYA DISEASE, MOYAMOYA SYNDROME AND INTIMAL PROLIFERATION
The present disclosure provides compositions and methods for treating allograft vasculopathy, for treating Moyamoya Diseases (MMD) and Moyamoya Syndrome (MMS), for treating inhibiting or preventing unwanted intimal proliferation in a subject by administering an ectonucleotide pyrophosphatase phosphodiesterase- 1 (ENPP1) agent or an ectonucleotide pyrophosphatase phosphodiesterase-3 (ENPP3).
A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
In certain aspects, the present invention provides novel soluble ENPP1 or ENPP3 polypeptides, as well as compositions and methods for using those variants to treat an indication associated an ENPP1 or ENPP3 deficiency. The compositions and methods provided herein are useful in treating diseases associated with an ENPP1 or ENPP3 deficiency such as pathological calcification or pathological ossification.
The present disclosure provides compositions and methods for treating Peripheral Artery Diseases in a subject by administering an ectonucleotide pyrophosphatase phosphodiesterase-1 (ENPP1) agent or an ectonucleotide pyrophosphatase phosphodiesterase-3 (ENPP3).
The present disclosure provides compositions and methods for treating vascular smooth muscle cell proliferation in a subject that does not have a deficiency of ectonucleotide pyrophosphatase phosphodiesterase- 1 (ENPP1) resulting in a pathological disease of calcification or ossification by administering an ENPP1 agent or an ENPP3 agent.
A61P 9/10 - Médicaments pour le traitement des troubles du système cardiovasculaire des maladies ischémiques ou athéroscléreuses, p. ex. médicaments antiangineux, vasodilatateurs coronariens, médicaments pour le traitement de l'infarctus du myocarde, de la rétinopathie, de l'insuffisance cérébro-vasculaire, de l'artériosclérose rénale
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
Pharmaceutical preparations for the diagnosis and
therapeutic treatment of diseases or disorders afflicting
the bone, kidney and tissue, diseases of abnormal
mineralization and calcification, diseases of the
circulatory system, diseases caused by inborn errors of
metabolism, and diseases caused by abnormal intimal
proliferation; pharmaceutical products for use in the
diagnosis and treatment of bone, kidney and tissue diseases,
diseases of abnormal mineralization and calcification,
diseases of the circulatory system, diseases caused by
inborn errors of metabolism, and diseases caused by abnormal
intimal proliferation.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
(1) Pharmaceutical preparations for the diagnosis and therapeutic treatment of bone and kidney diseases and disorders, tissue diseases in the nature of rickets, abnormal mineralization of vessels, intimal proliferation, chronic kidney disease (CKD), and skin calcification, diseases of abnormal mineralization and calcification, diseases of the circulatory system and diseases caused by inborn errors of metabolism in the nature of ENPP1 deficiency (GACI type I/ARHR2), ABCC6 deficiency (GACI type II, PXE), hypopyrophosphatemia, and diseases caused by abnormal intimal proliferation, none of the aforesaid pharmaceuticals for use in the treatment of Pompe Disease.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
therapeutic pharmaceutical for the diagnosis and treatment of diseases or disorders afflicting the bone, kidney and tissue, diseases of abnormal mineralization and calcification, diseases of the circulatory system, diseases caused by inborn errors of metabolism, and diseases caused by abnormal intimal proliferation; pharmaceutical products for use in the diagnosis and treatment of bone, kidney and tissue diseases, diseases of abnormal mineralization and calcification, diseases of the circulatory system, diseases caused by inborn errors of metabolism, and diseases caused by abnormal intimal proliferation
The present invention provides a method of treating myointimal proliferation by administering a recombinant human soluble ectonucleotide pyrophosphatase phosphodiesterase (hsNPP1), active fragment or fusion protein thereof.
A61P 9/10 - Médicaments pour le traitement des troubles du système cardiovasculaire des maladies ischémiques ou athéroscléreuses, p. ex. médicaments antiangineux, vasodilatateurs coronariens, médicaments pour le traitement de l'infarctus du myocarde, de la rétinopathie, de l'insuffisance cérébro-vasculaire, de l'artériosclérose rénale
C12N 9/16 - Hydrolases (3.) agissant sur les liaisons esters (3.1)
A01K 67/027 - Nouvelles races ou races modifiées de vertébrés
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
32.
Methods of improving cardiovascular function and treating cardiovascular disease using a recombinant ectonucleotide pyrophosphatase phosphodiesterase (NPP1)
The present invention provides methods for improving cardiovascular function in a human patient (e.g., reducing hypertension), as well as methods of treating a cardiovascular disease, by administering a recombinant human soluble ectonucleotide pyrophosphatase phosphodiesterase (hsNPP1), active fragment or fusion protein thereof.
C12N 9/16 - Hydrolases (3.) agissant sur les liaisons esters (3.1)
A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
A61P 9/10 - Médicaments pour le traitement des troubles du système cardiovasculaire des maladies ischémiques ou athéroscléreuses, p. ex. médicaments antiangineux, vasodilatateurs coronariens, médicaments pour le traitement de l'infarctus du myocarde, de la rétinopathie, de l'insuffisance cérébro-vasculaire, de l'artériosclérose rénale
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
Pharmaceutical preparations; diagnostic preparations for medical purposes; none of the aforesaid including pharmaceuticals for use in the treatment of pompe disease.
34.
TREATMENT OF DISEASES INVOLVING DEFICIENCY OF ENPP1 OR ENPP3
The present invention provides a method of treating NPP1 deficiency or NPP1-associated disease such as idiopathic infantile arterial calcification (IIAC), pseudoxanthoma elasticum, vascular calcification in chronic kidney disease (VCCKD), insulin resistance, hypophosphatemic rickets, myocardial ischemia, joint calcification, angioid streaks, and ossification of the posterior longitudinal ligament of the spine. The present invention provides a method for treating tissue calcification by administering soluble NPP1 to produce a transient increase in serum pyrophosphate levels.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
Pharmaceutical preparations, in particular therapeutic pharmaceuticals for the diagnosis and treatment of diseases afflicting the bone, kidney and tissue and pharmaceutical products for use in the diagnosis and treatment of bone, kidney and tissue diseases, none of the aforesaid including pharmaceuticals for use in the treatment of Pompe Disease.
05 - Produits pharmaceutiques, vétérinaires et hygièniques
Produits et services
(1) Pharmaceutical preparations and substances for use in the diagnosis and treatment of bone diseases; pharmaceutical preparations and substances for use in the diagnosis and treatment of kidney diseases and disorders; pharmaceutical preparations and substances for the treatment and prevention of abnormal mineralization and calcification disorders; pharmaceutical preparations and substances for use in the diagnosis and treatment of tissue diseases and disorders, namely bone disease (rickets), blood vessel (abnormal mineralization of vessels, Intimal Proliferation), kidney(CKD), Skin calcification; pharmaceutical preparations and substances for use in the diagnosis and treatment of circulatory diseases and disorders, namely ENPP1 Deficiency (GACI type I/ARHR2), ABCC6 Deficiency (GACI type II, PXE), Hypopyrophosphatemia and disease of abnormal mineralization of the heart, heart valves, blood vessels, skin, Kidney (hyperphosphaturia, nephrocalcinosis, tubular defects resulting from abnormal phosphorus handling); pharmaceutical preparations and substances for use in the diagnosis and treatment of inborn errors of metabolism, namely ENPP1 Deficiency, ABCC6 Deficiency, Hypopyrophosphatemia; pharmaceutical preparations and substances for use in the diagnosis and treatment of diseases caused by abnormal intimal proliferation; none of the aforesaid including pharmaceuticals for use in the treatment of Pompe disease.
The present invention provides a novel fusion polypeptide containing a catalytic portion of NPP1 fused to a targeting moiety, nucleic acids encoding the fusion polypeptide, a vector containing the nucleic acid integrated thereinto, a host cell transformed with the vector and pharmaceutical compositions comprising the fusion polypeptide.
C12N 9/96 - Stabilisation d'une enzyme par formation d'un adduct ou d'une compositionFormation de conjugaisons d'enzymes
C12N 15/00 - Techniques de mutation ou génie génétiqueADN ou ARN concernant le génie génétique, vecteurs, p. ex. plasmides, ou leur isolement, leur préparation ou leur purificationUtilisation d'hôtes pour ceux-ci
C12N 15/52 - Gènes codant pour des enzymes ou des proenzymes
C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
C12N 9/16 - Hydrolases (3.) agissant sur les liaisons esters (3.1)
C12N 9/00 - Enzymes, p. ex. ligases (6.)ProenzymesCompositions les contenantProcédés pour préparer, activer, inhiber, séparer ou purifier des enzymes
The present invention provides a novel fusion polypeptide containing a catalytic portion of NPP1 fused to a targeting moiety, nucleic acids encoding the fusion polypeptide, a vector containing the nucleic acid integrated thereinto, a host cell transformed with the vector and pharmaceutical compositions comprising the fusion polypeptide.
C12N 9/96 - Stabilisation d'une enzyme par formation d'un adduct ou d'une compositionFormation de conjugaisons d'enzymes
C12N 15/00 - Techniques de mutation ou génie génétiqueADN ou ARN concernant le génie génétique, vecteurs, p. ex. plasmides, ou leur isolement, leur préparation ou leur purificationUtilisation d'hôtes pour ceux-ci
C12N 15/52 - Gènes codant pour des enzymes ou des proenzymes
C12N 15/63 - Introduction de matériel génétique étranger utilisant des vecteursVecteurs Utilisation d'hôtes pour ceux-ciRégulation de l'expression
C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
